Rare Disease Report

NORD News for March - The Voice of the Community

MARCH 17, 2016
Guest post by NORD

NORD Partners with The Hole in the Wall Gang on Free Rare Disease Summer Family Camp
Applications Are Due By April 1 For This FREE Family Experience

NORD is proud to be working with The Hole in the Wall Gang Camp on that organization’s first-ever Summer Family Camp specifically for children affected by rare diseases and their families.

Established in 1988 by actor Paul Newman, The Hole in the Wall Gang Camp is dedicated to ensuring that every child, including those with serious medical challenges, can experience the transformational spirit and friendships that go hand-in-hand with camp.

The camp will take place at The Hole in the Wall Gang Camp in northeastern Connecticut on June 2-5. April 1 is the deadline to submit an application. Additional information is available on the NORD website, and application forms may be downloaded from the NORD site.

Families living in New England and other northeastern states who have children age 16 or younger affected by rare diseases are invited to apply. Participation for the entire family in the camp is free of charge, thanks to the generosity of sponsors and donors. Up to 25 families can be accommodated in this family camping experience.

Running for Rare Marathon Team to Expand and Transition to NORD

The Running for Rare Marathon team will transition to new governance under NORD and will expand its program of running to raise awareness and funds to benefit the rare disease community.  NORD will take over the program from Sanofi Genzyme, the specialty care global business unit of Sanofi, this year.

Running for Rare highlights the unmet and often heartbreaking needs of the nearly 1 in 10 Americans living with rare diseases – sadly, most of whom are children – and provides a way for people to get involved,” said NORD President and CEO Peter L. Saltonstall. “We are honored to have the support of such inspiring runners and individuals whose efforts directly help others.”

Funds raised by Running for Rare will pay for medical testing for individuals who have exhausted all possible avenues to receive a medical diagnosis and are in the process of applying to the National Institutes of Health (NIH) Undiagnosed Diseases Network (UDN). Applicants to this network will be referred to NORD by the NIH UDN centers when it is determined that they qualify for financial assistance.

“As a longstanding partner of NORD, Sanofi Genzyme has been proud to establish and organize the Running for Rare marathon team for the past eight years,” said David Meeker, MD, Executive Vice President and Head of Sanofi Genzyme. “Transitioning the Running for Rare team to NORD will provide new opportunities for growth through NORD’s unmatched national network.”

The team began as a grassroots effort of four Genzyme employees in 2008, growing to more than 100 runners globally in 2015.  Over the years, the team has raised nearly $750,000 for NORD and is committed to bringing awareness and support for all 7,000 rare diseases.

“The growth and excitement Running for Rare experienced last year set the stage for new avenues of community development and partnerships,” said Phil Maderia, Chairman of the Running for Rare Advisory Board. “We are excited to grow under NORD and broaden the relationships we’ve built throughout the rare disease community.”

The hallmark of Running for Rare is its Rare Community Partners Program, which connects runners with individuals living with rare diseases. Each runner has a patient partner, and the program provides a platform for sharing stories, raising awareness and connecting with others.

Running for Rare is currently accepting runners and Rare Community Partners for the Boston Marathon on April 18, Providence Full- and Half-Marathons on May 1, and New York City Marathon on May 6. For information, visit www.rarediseases.org/runningteam.


Erdheim-Chester Disease Scientific Symposium and Patient/Family Gathering are Planned
The 4th annual International ECD (Erdheim-Chester Disease) Scientific Symposium will take place in Paris on Sept. 15. A gathering for ECD patients and families will take place on Sept. 16, also in Paris. Visit the ECD Global Alliance website for details.

12th International VHL Medical Symposium to Take Place April 7-9
In collaboration with Massachusetts General Hospital, Boston University and the national Central University of Taiwan, the VHL (Von Hippel-Lindau) Alliance will host its biennial medical symposium in Boston at the Starr Center of the Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary. This event is CME-accredited by Boston University Medical Center. 

Research Symposium Brings Four Rare Childhood Disease Communities Together
A landmark national research symposium (June 21-24) and family conference (June 24-26) will draw together researchers and families to share their experiences with four childhood rare diseases: Rett syndrome, CDKL5 disorder,  FOXG1 disorder and MECP2 Duplication. To take place in suburban Chicago, this symposium has been developed with a theme of advancing knowledge and progress through collaboration.

Mucolipidosis Type IV Research and Family Conference Planned for June 19-21
Since 2013, the ML4 Foundation has sponsored an international conference on MLIV covering basic, clinical and translational research. The 2016 conference will take place in Atlanta June 19-21.

2016 Turner Syndrome National Conference to Take Place July 22-24 in Cincinnati
The 2016 National Turner Syndrome Conference will take place July 22-24 in Cincinnati. This event will include an International Turner Syndrome Clinical Care Guidelines Consensus Meeting. In addition, a camp for teens and young women with Turner syndrome will be held July 31-August 7.

14th Annual Patient Education Conference on Eosinophilic Gastrointestinal Disorders is Planned
The American Partnership for Eosinophilic Disorders (APFED) will host its 14th annual conference in San Diego July 8-9.

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